Dr. Vinay Prasad, head of the FDA's vaccines and biologics division, is stepping down at the end of April after a controversial year overseeing approvals for vaccines, gene therapies, and biotech products.
UniQure's stock falls sharply in premarket trading after the FDA says early data for AMT-130 isn't enough for approval. The company plans more talks for a Phase III trial on this Huntington's disease treatment.
uniQure shares fell about 33% after FDA Commissioner Marty Makary's comments on rare disease drugs. But analysts say his remarks likely targeted another therapy, not uniQure's AMT-130 for Huntington's disease.
AMT-191 gene therapy demonstrates sustained enzyme activity increases in Fabry disease patients, with 6 of 11 stopping traditional infusion therapy.
uniQure secures FDA meeting to discuss accelerated approval for AMT-130, bringing hope to the Huntington's disease community with potentially life-changing gene therapy.
UniQure stock tumbles as FDA says AMT-130 gene therapy data insufficient for approval. Company seeks urgent Q1 2026 meeting to advance Huntington's disease treatment.
